用于癌症过继性T细胞免疫疗法的基因组编辑技术
Genome-Editing Technologies in Adoptive T Cell Immunotherapy for Cancer.
作者信息
Singh Nathan, Shi Junwei, June Carl H, Ruella Marco
机构信息
Center for Cellular Immunotherapies, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.
Division of Hematology and Oncology, University of Pennsylvania, Philadelphia, PA, USA.
出版信息
Curr Hematol Malig Rep. 2017 Dec;12(6):522-529. doi: 10.1007/s11899-017-0417-7.
PURPOSE OF REVIEW
In this review, we discuss the most recent developments in gene-editing technology and discuss their application to adoptive T cell immunotherapy.
RECENT FINDINGS
Engineered T cell therapies targeting cancer antigens have demonstrated significant efficacy in specific patient populations. Most impressively, CD19-directed chimeric antigen receptor T cells (CART19) have led to impressive responses in patients with B-cell leukemia and lymphoma. CTL019, or KYMRIAH™ (tisagenlecleucel), a CD19 CAR T cell product developed by Novartis and the University of Pennsylvania, was recently approved for clinical use by the Food and Drug Administration, representing a landmark in the application of adoptive T cell therapies. As CART19 enters routine clinical use, improving the efficacy of this exciting platform is the next step in broader application. Novel gene-editing technologies like CRISPR-Cas9 allow facile editing of specific genes within the genome, generating a powerful platform to further optimize the activity of engineered T cells.
综述目的
在本综述中，我们讨论了基因编辑技术的最新进展，并探讨了其在过继性T细胞免疫治疗中的应用。
最新发现
靶向癌症抗原的工程化T细胞疗法在特定患者群体中已显示出显著疗效。最令人印象深刻的是，靶向CD19的嵌合抗原受体T细胞（CART19）在B细胞白血病和淋巴瘤患者中引发了令人瞩目的反应。CTL019，即诺华公司和宾夕法尼亚大学联合开发的CD19嵌合抗原受体T细胞产品KYMRIAH™（tisagenlecleucel），最近已获美国食品药品监督管理局批准用于临床，这代表了过继性T细胞疗法应用中的一个里程碑。随着CART19进入常规临床应用，提高这个令人振奋的平台的疗效是更广泛应用的下一步。像CRISPR-Cas9这样的新型基因编辑技术能够轻松编辑基因组内的特定基因，从而形成一个强大的平台，可进一步优化工程化T细胞的活性。
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